The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.

BH-30643 is an OMNI-EGFR inhibitor that targets classical, atypical, and compound EGFR mutations and acquired resistance mutations in HER2.

The companies will focus on advancing AXV-101, with which they hope to treat retinal dystrophy by targeting mutations in the BBS1 gene.

In a Phase I/II trial, patients with X-linked chronic granulomatous disease will receive a treatment created using a new ...

Doctors and patient advocates supporting pretreatment testing at times challenged FDA and other experts who aren't swayed by available evidence.

The company expects to submit a biologics license application for NTLA-2002 in 2026 and potentially launch it in the US in 2027.

The resolution aims to provide Illinois patients with equitable access to emerging treatments, including precision and genomic medicines.

The deal advances Galapagos' plan to develop a decentralized CAR T-cell therapy for non-Hodgkin lymphoma with a one-week vein-to-vein time.

HHS is asking the court to affirm its Office of Inspector General's finding that paying for fertility services would run ...

The company's multiple myeloma franchise, which includes Carvykti, stands to contribute significantly to its growth in 2025.

The Swedish firm has treated the first two patients in a Phase I/II trial of the autologous neoantigen-directed lymphocyte therapy.

The drug was previously available through the Cancer Drugs Fund, but with more data, NICE is backing routine access through the NHS.